Forward and Reverse Clinical Translation with RNA Aptamers

Dr. Bruce Sullenger’s research interests focuses on the development of RNA-based therapeutic agents for the potential treatment of cardiovascular diseases and for modulating immune responses. His group is also involved in advancing RNA-based tumor targeting strategies for delivering siRNAs and miRNAs to tumor cells. In particular, Bruce is a leader in the development of nucleic acid aptamers for inhibiting the activities of coagulation factors and platelet proteins. Dr. Sullenger co-founded Regado Biosciences Inc in 2001, to evaluate in particular the potential of an RNA aptamer termed Pegnivacogin [and its matched antidote] to treat patients with acute coronary syndrome in clinical trial studies. Pegnivacogin inhibits the action of coagulation factor IXa. His company merged with Tobira Therapeutics. His talk today will highlight the discovery path and challenges facing the clinical translation of RNA aptamers to the clinic.